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Open-label Phase 3 Study With Mirabegron in Children From 3 to Less Than 18 Years of Age With Neurogenic Detrusor Overactivity (Crocodile)

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ClinicalTrials.gov Identifier: NCT02751931
Recruitment Status : Completed
First Posted : April 26, 2016
Results First Posted : May 12, 2020
Last Update Posted : May 12, 2020
Sponsor:
Information provided by (Responsible Party):
Astellas Pharma Inc ( Astellas Pharma Europe B.V. )

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Neurogenic Detrusor Overactivity
Intervention Drug: Mirabegron
Enrollment 91
Recruitment Details Pediatric participants consisting of male and female children from 3 to <12 years of age and adolescents from 12 to <18 years of age, with a body weight of >=11 kilogram (kg), with Neurogenic detrusor overactivity (NDO) on clean intermittent catheterization (CIC) were enrolled in this study.
Pre-assignment Details Eligible participants who met inclusion and none of the exclusion criteria were enrolled. Participants who received oral drug to manage their NDO completed a 2 week washout period. A total of 113 pediatric participants were screened, 22 of whom were screening failures.
Arm/Group Title Children (3 to < 12 Years) Adolescents (12 to < 18 Years)
Hide Arm/Group Description Participants aged 3 to < 12 years received initial dose of 25 milligram (mg) of mirabegron orally once daily based on weight (pediatric equivalent dose of 25 mg (milligram) [PED25]) on day 1. At weeks 2, 4 or 8, participant's were up-titrated to the pediatric equivalent dose of 50 mg in adults [PED50] based on the given dose titration criteria. Following week 24, participants stayed on their individual dose level until week 52 end-of-study (EOS) or end-of-treatment (EOT). Participants aged 12 to < 18 years received initial dose of 25 mg of mirabegron orally once daily based on weight [PED25] on day 1. At weeks 2, 4 or 8, participant's were up-titrated to the pediatric equivalent dose of 50 mg in adults [PED50] based on the given dose titration criteria. Following week 24, participants stayed on their individual dose level until week 52 EOS or EOT.
Period Title: Overall Study
Started 56 35
Treated 55 31
Completed 43 27
Not Completed 13 8
Reason Not Completed
Adverse Event             3             0
Other: Miscelleneous             10             8
Arm/Group Title Children (3 to < 12 Years) Adolescents (12 to < 18 Years) Total
Hide Arm/Group Description Participants aged 3 to < 12 years received initial dose of 25 mg of mirabegron orally once daily based on weight [PED25] on day 1. At weeks 2, 4 or 8, participant's were up-titrated to the pediatric equivalent dose of 50 mg in adults [PED50] based on the given dose titration criteria. Following week 24, participants stayed on their individual dose level until week 52 EOS or EOT. Participants aged 12 to < 18 years received initial dose of 25 mg of mirabegron orally once daily based on weight [PED25] on day 1. At weeks 2, 4 or 8, participant's were up-titrated to the pediatric equivalent dose of 50 mg in adults [PED50] based on the given dose titration criteria. Following week 24, participants stayed on their individual dose level until week 52 EOS or EOT. Total of all reporting groups
Overall Number of Baseline Participants 56 35 91
Hide Baseline Analysis Population Description
All Allocated Set included all participants who had a non-missing registration date at visit 3 (baseline).
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 56 participants 35 participants 91 participants
7.9  (2.5) 13.9  (1.6) 10.2  (3.7)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 56 participants 35 participants 91 participants
Female
33
  58.9%
15
  42.9%
48
  52.7%
Male
23
  41.1%
20
  57.1%
43
  47.3%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 56 participants 35 participants 91 participants
Hispanic or Latino
1
   1.8%
2
   5.7%
3
   3.3%
Not Hispanic or Latino
55
  98.2%
33
  94.3%
88
  96.7%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 56 participants 35 participants 91 participants
American Indian or Alaska Native
0
   0.0%
1
   2.9%
1
   1.1%
Asian
13
  23.2%
8
  22.9%
21
  23.1%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
0
   0.0%
White
41
  73.2%
25
  71.4%
66
  72.5%
More than one race
2
   3.6%
1
   2.9%
3
   3.3%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
Maximum Cystometric Capacity   [1] [2] 
Mean (Standard Deviation)
Unit of measure:  Milliliter (mL)
Number Analyzed 43 participants 25 participants 68 participants
158.64  (94.50) 238.92  (99.14) 188.16  (103.15)
[1]
Measure Description: MCC was defined as the maximum bladder capacity reached during filling cystometry before either leakage or pain/discomfort was observed.
[2]
Measure Analysis Population Description: The analysis population consisted of the FAS (full analysis set) which included all participants who took >= 1 dose of study drug and provided both valid (as by the central reviewer’s assessment) nonmissing MCC measurements at baseline and at a postbaseline visit for the primary efficacy endpoint.
1.Primary Outcome
Title Change From Baseline in Maximum Cystometric Capacity (MCC) at Week 24
Hide Description Change from baseline in MCC was based on filling urodynamics (volume at the end of filling). During urodynamic assessments, the bladder was filled until voiding/leakage began, or until the participant experienced pain or discomfort, or because dangerous high detrusor pressure, or 135% of maximum catheterized volume for age had been reached. A valid urodynamic assessment was confirmed valid by the central reviewers. Missing MCC observations at week 24 were imputed using last observation carried forward (LOCF).
Time Frame Baseline and week 24
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis population consisted of the full analysis set (FAS) which included all participants who took ≥ 1 dose of study drug and provided both valid (as by the central reviewer’s assessment) nonmissing MCC measurements at baseline and at a postbaseline visit for the primary efficacy endpoint.
Arm/Group Title Children (3 to < 12 Years) Adolescents (12 to < 18 Years)
Hide Arm/Group Description:
Participants aged 3 to < 12 years received initial dose of 25 mg of mirabegron orally once daily based on weight [PED25] on day 1. At weeks 2, 4 or 8, participant's were up-titrated to the pediatric equivalent dose of 50 mg in adults (PED50) based on the given dose titration criteria. Following week 24, participants stayed on their individual dose level until week 52 EOS or EOT.
Participants aged 12 to < 18 years received initial dose of 25 mg of mirabegron orally once daily based on weight [PED25] on day 1. At weeks 2, 4 or 8, participant's were up-titrated to the pediatric equivalent dose of 50 mg in adults (PED50) based on the given dose titration criteria. Following week 24, participants stayed on their individual dose level until week 52 EOS or EOT.
Overall Number of Participants Analyzed 43 25
Mean (Standard Deviation)
Unit of Measure: mL
72.09  (87.09) 113.21  (82.99)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 24 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 72.09
Confidence Interval (2-Sided) 95%
45.28 to 98.89
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 24 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 113.21
Confidence Interval (2-Sided) 95%
78.95 to 147.47
Estimation Comments [Not Specified]
2.Secondary Outcome
Title Change From Baseline in Bladder Compliance (ΔV/ΔP)
Hide Description Bladder compliance was an indication of the elasticity of the bladder wall and was calculated by dividing the change in volume by the change in detrusor pressure during the filling of the bladder. Change from baseline in bladder compliance (change in volume/change in pressure) was assessed by the independent central reviewers and reported as annotations on the urodynamic trace and in an external database. During urodynamic assessments, the bladder was filled until voiding/leakage began, or until the participant experienced pain or discomfort, or because dangerous high detrusor pressure, or 135% of maximum catheterized volume for age had been reached. A valid urodynamic assessment was confirmed valid by the central reviewers.
Time Frame Baseline and weeks 4 and 24
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis population consisted of the FAS. Here, Overall Number of participants analyzed signifies number of participants evaluable for this outcome measure and number analyzed signifies number of participants with available data at each time point.
Arm/Group Title Children (3 to < 12 Years) Adolescents (12 to < 18 Years)
Hide Arm/Group Description:
Participants aged 3 to < 12 years received initial dose of 25 mg of mirabegron orally once daily based on weight [PED25] on day 1. At weeks 2, 4 or 8, participant's were up-titrated to the pediatric equivalent dose of 50 mg in adults [PED50] based on the given dose titration criteria. Following week 24, participants stayed on their individual dose level until week 52 EOS or EOT.
Participants aged 12 to < 18 years received initial dose of 25 mg of mirabegron orally once daily based on weight [PED25] on day 1. At weeks 2, 4 or 8, participant's were up-titrated to the pediatric equivalent dose of 50 mg in adults [PED50] based on the given dose titration criteria. Following week 24, participants stayed on their individual dose level until week 52 EOS or EOT.
Overall Number of Participants Analyzed 39 22
Mean (Standard Deviation)
Unit of Measure: mL/cm H2O
Change at Week 4 Number Analyzed 39 participants 22 participants
-4.09  (50.78) 15.16  (22.69)
Change at Week 24 Number Analyzed 33 participants 21 participants
14.62  (42.09) 13.59  (15.02)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 4 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.618
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -4.09
Confidence Interval (2-Sided) 95%
-20.55 to 12.38
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 4 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.005
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 15.16
Confidence Interval (2-Sided) 95%
5.10 to 25.22
Estimation Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 24 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.055
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 14.62
Confidence Interval (2-Sided) 95%
-0.31 to 29.54
Estimation Comments [Not Specified]
Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 24 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 13.59
Confidence Interval (2-Sided) 95%
6.75 to 20.42
Estimation Comments [Not Specified]
3.Secondary Outcome
Title Change From Baseline in Maximum Cystometric Capacity at Week 4
Hide Description Change from baseline in MCC was based on filling urodynamics (volume at the end of filling). During urodynamic assessments, the bladder was filled until voiding/leakage began, or until the participant experienced pain or discomfort, or because dangerous high detrusor pressure, or 135% of maximum catheterized volume for age had been reached. A valid urodynamic assessment was confirmed valid by the central reviewers.
Time Frame Baseline and week 4
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis population consisted of the FAS. Here, Overall Number of participants analyzed signifies those who where evaluable for this outcome measure.
Arm/Group Title Children (3 to < 12 Years) Adolescents (12 to < 18 Years)
Hide Arm/Group Description:
Participants aged 3 to < 12 years received initial dose of 25 mg of mirabegron orally once daily based on weight [PED25] on day 1. At weeks 2, 4 or 8, participant's were up-titrated to the pediatric equivalent dose of 50 mg in adults (PED50) based on the given dose titration criteria. Following week 24, participants stayed on their individual dose level until week 52 EOS or EOT.
Participants aged 12 to < 18 years received initial dose of 25 mg of mirabegron orally once daily based on weight [PED25] on day 1. At weeks 2, 4 or 8, participant's were up-titrated to the pediatric equivalent dose of 50 mg in adults (PED50) based on the given dose titration criteria. Following week 24, participants stayed on their individual dose level until week 52 EOS or EOT.
Overall Number of Participants Analyzed 41 23
Mean (Standard Deviation)
Unit of Measure: mL
41.36  (71.64) 80.78  (96.15)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change from Baseline at week 4 - Children.
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 41.36
Confidence Interval (2-Sided) 95%
18.75 to 63.97
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 4 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 80.78
Confidence Interval (2-Sided) 95%
39.20 to 122.36
Estimation Comments [Not Specified]
4.Secondary Outcome
Title Change From Baseline in Number of Overactive Detrusor Contractions (> 15 cm H20) Until End of Filling
Hide Description Detrusor overactivity is the occurrence of involuntary detrusor contractions during filling cystometry. During urodynamic assessments, the bladder was filled until voiding/leakage began, or until the participant experienced pain or discomfort, or because dangerous high detrusor pressure, or 135% of maximum catheterized volume for age had been reached. A valid urodynamic assessment was confirmed valid by the central reviewers.
Time Frame Baseline and weeks 4 and 24
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis population consisted of the FAS. Here, Overall Number of participants analyzed signifies number of participants evaluable for this outcome measure and number analyzed signifies number of participants with available data at each time point.
Arm/Group Title Children (3 to < 12 Years) Adolescents (12 to < 18 Years)
Hide Arm/Group Description:
Participants aged 3 to < 12 years received initial dose of 25 mg of mirabegron orally once daily based on weight [PED25] on day 1. At weeks 2, 4 or 8, participant's were up-titrated to the pediatric equivalent dose of 50 mg in adults [PED50] based on the given dose titration criteria. Following week 24, participants stayed on their individual dose level until week 52 EOS or EOT.
Participants aged 12 to < 18 years received initial dose of 25 mg of mirabegron orally once daily based on weight [PED25] on day 1. At weeks 2, 4 or 8, participant's were up-titrated to the pediatric equivalent dose of 50 mg in adults [PED50] based on the given dose titration criteria. Following week 24, participants stayed on their individual dose level until week 52 EOS or EOT.
Overall Number of Participants Analyzed 41 22
Mean (Standard Deviation)
Unit of Measure: overactive detrusor contractions
Change at Week 4 Number Analyzed 41 participants 22 participants
0.44  (5.82) -0.64  (2.94)
Change at Week 24 Number Analyzed 36 participants 22 participants
-1.86  (4.16) -0.77  (3.87)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 4 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.632
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 0.44
Confidence Interval (2-Sided) 95%
-1.40 to 2.28
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 4 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.321
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -0.64
Confidence Interval (2-Sided) 95%
-1.94 to 0.67
Estimation Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 24 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.011
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -1.86
Confidence Interval (2-Sided) 95%
-3.27 to -0.45
Estimation Comments [Not Specified]
Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 24 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.359
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -0.77
Confidence Interval (2-Sided) 95%
-2.49 to 0.94
Estimation Comments [Not Specified]
5.Secondary Outcome
Title Change From Baseline in Detrusor Pressure at End of Filling
Hide Description Detrusor pressure was defined as bladder pressure minus intra-abdominal pressure as assessed by urodynamics. Filling was stopped (end of filling) when the detrusor pressure exceeded 100 cm H2O or was considered dangerously high by the investigator or urodynamicist (for instance, a prolonged passive detrusor pressure > 40 cm H2O). During urodynamic assessments, the bladder was filled until voiding/leakage began, or until the participant experienced pain or discomfort, or because dangerous high detrusor pressure, or 135% of maximum catheterized volume for age had been reached. A valid urodynamic assessment was confirmed valid by the central reviewers.
Time Frame Baseline and weeks 4 and 24
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis population consisted of the FAS. Here, Overall Number of participants analyzed signifies number of participants evaluable for this outcome measure and number analyzed signifies number of participants with available data at each time point.
Arm/Group Title Children (3 to < 12 Years) Adolescents (12 to < 18 Years)
Hide Arm/Group Description:
Participants aged 3 to < 12 years received initial dose of 25 mg of mirabegron orally once daily based on weight [PED25] on day 1. At weeks 2, 4 or 8, participant's were up-titrated to the pediatric equivalent dose of 50 mg in adults [PED50] based on the given dose titration criteria. Following week 24, participants stayed on their individual dose level until week 52 EOS or EOT.
Participants aged 12 to < 18 years received initial dose of 25 mg of mirabegron orally once daily based on weight [PED25] on day 1. At weeks 2, 4 or 8, participant's were up-titrated to the pediatric equivalent dose of 50 mg in adults [PED50] based on the given dose titration criteria. Following week 24, participants stayed on their individual dose level until week 52 EOS or EOT.
Overall Number of Participants Analyzed 41 22
Mean (Standard Deviation)
Unit of Measure: cm H2O
Change at Week 4 Number Analyzed 41 participants 22 participants
-12.38  (19.56) -6.48  (30.70)
Change at Week 24 Number Analyzed 36 participants 22 participants
-18.11  (19.97) -13.19  (19.91)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 4 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -12.38
Confidence Interval (2-Sided) 95%
-18.56 to -6.21
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 4 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.334
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -6.48
Confidence Interval (2-Sided) 95%
-20.09 to 7.13
Estimation Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 24 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -18.11
Confidence Interval (2-Sided) 95%
-24.87 to -11.35
Estimation Comments [Not Specified]
Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 24 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.005
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -13.19
Confidence Interval 95%
-22.02 to -4.36
Estimation Comments [Not Specified]
6.Secondary Outcome
Title Change From Baseline in Filling Bladder Volume Until First Overactive Detrusor Contraction (> 15 cm H20)
Hide Description Detrusor overactivity is the occurrence of involuntary detrusor contractions during filling cystometry. During urodynamic assessments, the bladder was filled until voiding/leakage began, or until the participant experienced pain or discomfort, or because dangerous high detrusor pressure, or 135% of maximum catheterized volume for age had been reached. A valid urodynamic assessment was confirmed valid by the central reviewers. If no detrusor contraction of > 15 cm H2O occurred, the bladder volume was imputed with maximum cystometric capacity.
Time Frame Baseline and weeks 4 and 24
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis population consisted of the FAS. Here, Overall Number of participants analyzed signifies number of participants evaluable for this outcome measure and number analyzed signifies number of participants with available data at each time point.
Arm/Group Title Children (3 to < 12 Years) Adolescents (12 to < 18 Years)
Hide Arm/Group Description:
Participants aged 3 to < 12 years received initial dose of 25 mg of mirabegron orally once daily based on weight [PED25] on day 1. At weeks 2, 4 or 8, participant's were up-titrated to the pediatric equivalent dose of 50 mg in adults [PED50] based on the given dose titration criteria. Following week 24, participants stayed on their individual dose level until week 52 EOS or EOT.
Participants aged 12 to < 18 years received initial dose of 25 mg of mirabegron orally once daily based on weight [PED25] on day 1. At weeks 2, 4 or 8, participant's were up-titrated to the pediatric equivalent dose of 50 mg in adults [PED50] based on the given dose titration criteria. Following week 24, participants stayed on their individual dose level until week 52 EOS or EOT.
Overall Number of Participants Analyzed 21 8
Median (Inter-Quartile Range)
Unit of Measure: mL
Change at Week 4 Number Analyzed 21 participants 8 participants
54.00
(13.00 to 105.30)
41.15
(3.00 to 62.50)
Change at Week 24 Number Analyzed 13 participants 8 participants
68.00
(32.00 to 110.0)
62.00
(4.00 to 95.15)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 4 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments From a Wilcoxon signed-rank test, testing the null hypothesis that week 4 median is equal to baseline median.
Method Wilcoxon signed-rank test
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 4 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.148
Comments From a Wilcoxon signed-rank test, testing the null hypothesis that week 4 median is equal to baseline median.
Method Wilcoxon signed-rank test
Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 24 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.002
Comments From a Wilcoxon signed-rank test, testing the null hypothesis that week 24 median is equal to baseline median.
Method Wilcoxon signed-rank test
Comments [Not Specified]
Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 24 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.039
Comments From a Wilcoxon signed-rank test, testing the null hypothesis that week 24 median is equal to baseline median.
Method Wilcoxon signed-rank test
Comments [Not Specified]
7.Secondary Outcome
Title Change From Baseline in Average Catheterized Volume Per Catheterization
Hide Description For each participant, the average catheterized volume per catheterization was calculated as the sum of all available/non-missing catheterized volumes recorded over 2 measuring days in the weekend diary, whether or not the 2 days were consecutive divided by the number of catheterizations with non-missing volumes. If volumes were recorded on 1 single day of the weekend diary, the average catheterized volume per catheterization was calculated using all available/non-missing catheterized volumes recorded that day. If no volumes were recorded on any day of the weekend diary, the average catheterized volume per catheterization was missing. A valid bladder diary day in the weekend diary was any e-diary day for which ≥1 catheterized volume >0 mL was recorded with complete date and time.
Time Frame Baseline and weeks 2, 4, 8, 12, 24, 36 and 52
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis population consisted of the FAS. Here, Overall Number of participants analyzed signifies number of participants evaluable for this outcome measure and number analyzed signifies number of participants with available data at each time point.
Arm/Group Title Children (3 to < 12 Years) Adolescents (12 to < 18 Years)
Hide Arm/Group Description:
Participants aged 3 to < 12 years received initial dose of 25 mg of mirabegron orally once daily based on weight [PED25] on day 1. At weeks 2, 4 or 8, participant's were up-titrated to the pediatric equivalent dose of 50 mg in adults [PED50] based on the given dose titration criteria. Following week 24, participants stayed on their individual dose level until week 52 EOS or EOT.
Participants aged 12 to < 18 years received initial dose of 25 mg of mirabegron orally once daily based on weight [PED25] on day 1. At weeks 2, 4 or 8, participant's were up-titrated to the pediatric equivalent dose of 50 mg in adults [PED50] based on the given dose titration criteria. Following week 24, participants stayed on their individual dose level until week 52 EOS or EOT.
Overall Number of Participants Analyzed 43 24
Mean (Standard Deviation)
Unit of Measure: mL
Change at Week 2 Number Analyzed 43 participants 24 participants
14.58  (43.98) 35.99  (54.19)
Change at Week 4 Number Analyzed 42 participants 24 participants
30.08  (49.50) 51.96  (64.71)
Change at Week 8 Number Analyzed 43 participants 22 participants
36.90  (46.05) 45.10  (53.77)
Change at Week 12 Number Analyzed 43 participants 24 participants
32.25  (45.51) 43.94  (58.49)
Change at Week 24 Number Analyzed 41 participants 23 participants
41.63  (58.03) 59.31  (82.22)
Change at Week 36 Number Analyzed 40 participants 24 participants
53.87  (91.74) 52.14  (74.90)
Change at Week 52 Number Analyzed 40 participants 23 participants
42.84  (65.31) 42.40  (69.25)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 2 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.035
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 14.58
Confidence Interval (2-Sided) 95%
1.0 to 28.1
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 2 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.003
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 35.99
Confidence Interval (2-Sided) 95%
13.1 to 58.9
Estimation Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 4 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 30.08
Confidence Interval (2-Sided) 95%
14.7 to 45.5
Estimation Comments [Not Specified]
Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 4 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 51.96
Confidence Interval (2-Sided) 95%
24.6 to 79.3
Estimation Comments [Not Specified]
Hide Statistical Analysis 5
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 8 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 36.90
Confidence Interval (2-Sided) 95%
22.7 to 51.1
Estimation Comments [Not Specified]
Hide Statistical Analysis 6
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 8 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 45.10
Confidence Interval (2-Sided) 95%
21.3 to 68.9
Estimation Comments [Not Specified]
Hide Statistical Analysis 7
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 12 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 32.25
Confidence Interval (2-Sided) 95%
18.2 to 46.3
Estimation Comments [Not Specified]
Hide Statistical Analysis 8
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 12 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.001
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 43.94
Confidence Interval (2-Sided) 95%
19.2 to 68.6
Estimation Comments [Not Specified]
Hide Statistical Analysis 9
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 24 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 41.63
Confidence Interval (2-Sided) 95%
23.3 to 60.0
Estimation Comments [Not Specified]
Hide Statistical Analysis 10
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 24 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.002
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 59.31
Confidence Interval (2-Sided) 95%
23.8 to 94.9
Estimation Comments [Not Specified]
Hide Statistical Analysis 11
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 36 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 53.87
Confidence Interval (2-Sided) 95%
24.5 to 83.2
Estimation Comments [Not Specified]
Hide Statistical Analysis 12
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 36 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.002
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 52.14
Confidence Interval (2-Sided) 95%
20.5 to 83.8
Estimation Comments [Not Specified]
Hide Statistical Analysis 13
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 52 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 42.84
Confidence Interval (2-Sided) 95%
22.0 to 63.7
Estimation Comments [Not Specified]
Hide Statistical Analysis 14
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 52 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.008
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 42.40
Confidence Interval (2-Sided) 95%
12.5 to 72.3
Estimation Comments [Not Specified]
8.Secondary Outcome
Title Change From Baseline in Maximum Catheterized Volume
Hide Description For each participant, the maximum catheterized volume per day was calculated using all available/non-missing catheterized volumes recorded for the 2 measuring days in the weekend e-diary, whether or not these 2 days were consecutive. Maximum value was calculated separately for each measuring day and the mean of the two values was used. If volumes recorded on 1 single day of the weekend e-diary, the maximum catheterized volume per day was calculated using all available/non-zero catheterized volumes recorded that day. If no volumes were recorded on any day of the weekend e-diary, the maximum catheterized volume per day was missing. A valid bladder diary day in the weekend diary was any e-diary day for which >=1 catheterized volume >0 mL was recorded with complete date and time.
Time Frame Baseline and weeks 2, 4, 8, 12, 24, 36 and 52
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis population consisted of the FAS. Here, Overall Number of participants analyzed signifies number of participants evaluable for this outcome measure and number analyzed signifies number of participants with available data at each time point.
Arm/Group Title Children (3 to < 12 Years) Adolescents (12 to < 18 Years)
Hide Arm/Group Description:
Participants aged 3 to < 12 years received initial dose of 25 mg of mirabegron orally once daily based on weight [PED25] on day 1. At weeks 2, 4 or 8, participant's were up-titrated to the pediatric equivalent dose of 50 mg in adults [PED50] based on the given dose titration criteria. Following week 24, participants stayed on their individual dose level until week 52 EOS or EOT.
Participants aged 12 to < 18 years received initial dose of 25 mg of mirabegron orally once daily based on weight [PED25] on day 1. At weeks 2, 4 or 8, participant's were up-titrated to the pediatric equivalent dose of 50 mg in adults [PED50] based on the given dose titration criteria. Following week 24, participants stayed on their individual dose level until week 52 EOS or EOT.
Overall Number of Participants Analyzed 43 24
Mean (Standard Deviation)
Unit of Measure: mL
Change at Week 2 Number Analyzed 43 participants 24 participants
17.50  (73.58) 42.38  (78.23)
Change at Week 4 Number Analyzed 42 participants 24 participants
46.69  (80.29) 73.25  (103.98)
Change at Week 8 Number Analyzed 43 participants 22 participants
45.27  (75.22) 42.86  (79.97)
Change at Week 12 Number Analyzed 43 participants 24 participants
33.23  (68.31) 47.29  (69.83)
Change at Week 24 Number Analyzed 41 participants 23 participants
49.88  (103.70) 84.39  (121.98)
Change at Week 36 Number Analyzed 40 participants 24 participants
60.09  (121.66) 54.78  (104.54)
Change at Week 52 Number Analyzed 40 participants 23 participants
53.51  (96.72) 54.30  (104.74)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 2 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.126
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 17.50
Confidence Interval (2-Sided) 95%
-5.1 to 40.1
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 2 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.014
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 42.38
Confidence Interval (2-Sided) 95%
9.3 to 75.4
Estimation Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 4 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 46.69
Confidence Interval (2-Sided) 95%
21.7 to 71.7
Estimation Comments [Not Specified]
Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 4 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.002
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 73.25
Confidence Interval (2-Sided) 95%
29.3 to 117.2
Estimation Comments [Not Specified]
Hide Statistical Analysis 5
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 8 - Children
Type of Statistical Test Other
Comments Pre-Specified
Statistical Test of Hypothesis P-Value <0.001
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 45.27
Confidence Interval (2-Sided) 95%
22.1 to 68.4
Estimation Comments [Not Specified]
Hide Statistical Analysis 6
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 8 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.020
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 42.86
Confidence Interval (2-Sided) 95%
7.4 to 78.3
Estimation Comments [Not Specified]
Hide Statistical Analysis 7
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 12 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.003
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 33.23
Confidence Interval (2-Sided) 95%
12.2 to 54.3
Estimation Comments [Not Specified]
Hide Statistical Analysis 8
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 12 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.003
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 47.29
Confidence Interval (2-Sided) 95%
17.8 to 76.8
Estimation Comments [Not Specified]
Hide Statistical Analysis 9
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 24 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.004
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 49.88
Confidence Interval (2-Sided) 95%
17.1 to 82.6
Estimation Comments [Not Specified]
Hide Statistical Analysis 10
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 24 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.003
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 84.39
Confidence Interval (2-Sided) 95%
31.6 to 137.1
Estimation Comments [Not Specified]
Hide Statistical Analysis 11
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 36 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.003
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 60.09
Confidence Interval (2-Sided) 95%
21.2 to 99.0
Estimation Comments [Not Specified]
Hide Statistical Analysis 12
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 36 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.017
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 54.78
Confidence Interval (2-Sided) 95%
10.6 to 98.9
Estimation Comments [Not Specified]
Hide Statistical Analysis 13
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 52 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.001
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 53.51
Confidence Interval (2-Sided) 95%
22.6 to 84.4
Estimation Comments [Not Specified]
Hide Statistical Analysis 14
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 52 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.021
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 54.30
Confidence Interval (2-Sided) 95%
9.0 to 99.6
Estimation Comments [Not Specified]
9.Secondary Outcome
Title Change From Baseline in Maximum Catheterized Daytime Volume (MCDV)
Hide Description For each participant, the MCDV was calculated using all available/non-missing catheterized daytime volumes for the 2 measuring days in the weekend e-diary, whether or not the 2 days were consecutive. Maximum value was calculated separately for each measuring day and the mean of the 2 values was used. If volumes were recorded on 1 single day of the weekend e-diary, the MCDV was calculated using all available/non-zero catheterized daytime volumes recorded that day. If no volumes were recorded on any day of the weekend e-diary, the MCDV was missing. Daytime was defined as the time between wake-up time (minus 30 min) & time to sleep (plus 29 min) recorded in the e-diary. A valid bladder diary day in the weekend diary was any e-diary day for which >=1 catheterized volume >0 mL was recorded with complete date and time.
Time Frame Baseline and weeks 2, 4, 8, 12, 24, 36 and 52
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis population consisted of the FAS. Here, Overall Number of participants analyzed signifies number of participants evaluable for this outcome measure and number analyzed signifies number of participants with available data at each time point.
Arm/Group Title Children (3 to < 12 Years) Adolescents (12 to < 18 Years)
Hide Arm/Group Description:
Participants aged 3 to < 12 years received initial dose of 25 mg of mirabegron orally once daily based on weight [PED25] on day 1. At weeks 2, 4 or 8, participant's were up-titrated to the pediatric equivalent dose of 50 mg in adults [PED50] based on the given dose titration criteria. Following week 24, participants stayed on their individual dose level until week 52 EOS or EOT.
Participants aged 12 to < 18 years received initial dose of 25 mg of mirabegron orally once daily based on weight [PED25] on day 1. At weeks 2, 4 or 8, participant's were up-titrated to the pediatric equivalent dose of 50 mg in adults [PED50] based on the given dose titration criteria. Following week 24, participants stayed on their individual dose level until week 52 EOS or EOT.
Overall Number of Participants Analyzed 43 24
Mean (Standard Deviation)
Unit of Measure: mL
Change at Week 2 Number Analyzed 43 participants 24 participants
18.13  (73.38) 35.58  (86.78)
Change at Week 4 Number Analyzed 42 participants 24 participants
37.71  (83.33) 70.35  (113.98)
Change at Week 8 Number Analyzed 43 participants 22 participants
43.91  (74.44) 38.11  (90.88)
Change at Week 12 Number Analyzed 43 participants 24 participants
29.05  (67.86) 43.04  (73.82)
Change at Week 24 Number Analyzed 41 participants 23 participants
44.20  (98.31) 81.37  (117.77)
Change at Week 36 Number Analyzed 40 participants 24 participants
58.49  (121.12) 50.90  (114.05)
Change at Week 52 Number Analyzed 40 participants 23 participants
53.76  (100.24) 49.13  (117.23)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 2 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.113
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 18.13
Confidence Interval (2-Sided) 95%
-4.5 to 40.7
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 2 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.056
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 35.58
Confidence Interval (2-Sided) 95%
-1.1 to 72.2
Estimation Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 4 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.005
Comments [Not Specified]
Method t-test, 2 sided
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 37.71
Confidence Interval (2-Sided) 95%
11.7 to 63.7
Estimation Comments [Not Specified]
Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 4 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.006
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 70.35
Confidence Interval (2-Sided) 95%
22.2 to 118.5
Estimation Comments [Not Specified]
Hide Statistical Analysis 5
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 8 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 43.91
Confidence Interval (2-Sided) 95%
21.0 to 66.8
Estimation Comments [Not Specified]
Hide Statistical Analysis 6
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 8 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.063
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 38.11
Confidence Interval (2-Sided) 95%
-2.2 to 78.4
Estimation Comments [Not Specified]
Hide Statistical Analysis 7
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 12 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.008
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 29.05
Confidence Interval (2-Sided) 95%
8.2 to 49.9
Estimation Comments [Not Specified]
Hide Statistical Analysis 8
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 12 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.009
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 43.04
Confidence Interval (2-Sided) 95%
11.9 to 74.2
Estimation Comments [Not Specified]
Hide Statistical Analysis 9
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 24 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.006
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 44.20
Confidence Interval (2-Sided) 95%
13.2 to 75.2
Estimation Comments [Not Specified]
Hide Statistical Analysis 10
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 24 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.003
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 81.37
Confidence Interval (2-Sided) 95%
30.4 to 132.3
Estimation Comments [Not Specified]
Hide Statistical Analysis 11
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 36 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.004
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 58.49
Confidence Interval (2-Sided) 95%
19.8 to 97.2
Estimation Comments [Not Specified]
Hide Statistical Analysis 12
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 36 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.039
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 50.90
Confidence Interval (2-Sided) 95%
2.7 to 99.1
Estimation Comments [Not Specified]
Hide Statistical Analysis 13
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 52 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.002
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 53.76
Confidence Interval (2-Sided) 95%
21.7 to 85.8
Estimation Comments [Not Specified]
Hide Statistical Analysis 14
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 52 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.057
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 49.13
Confidence Interval (2-Sided) 95%
-1.6 to 99.8
Estimation Comments [Not Specified]
10.Secondary Outcome
Title Change From Baseline in Average Morning Catheterized Volume
Hide Description The first morning catheterized volume was the first recorded non-zero volume within or after the hour of the wake-up time on a volume-measuring day in the e-diary. The average first morning catheterized volume was calculated as the average of the available first morning catheterized volumes recorded for the 2 measuring days in the weekend e-diary, whether or not these 2 days were consecutive. If the first morning catheterized volume was recorded on 1 single day of the weekend e-diary, the average morning catheterized is the first morning catheterized that day. If no first morning catheterized volumes are recorded on any day of the weekend e-diary, the average first morning catheterized volume was missing. A valid bladder diary day in the weekend diary was any e-diary day for which >=1 catheterized volume >0 mL was recorded with complete date and time.
Time Frame Baseline and weeks 2, 4, 8, 12, 24, 36 and 52
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis population consisted of the FAS. Here, Overall Number of participants analyzed signifies number of participants evaluable for this outcome measure and number analyzed signifies number of participants with available data at each time point.
Arm/Group Title Children (3 to < 12 Years) Adolescents (12 to < 18 Years)
Hide Arm/Group Description:
Participants aged 3 to < 12 years received initial dose of 25 mg of mirabegron orally once daily based on weight [PED25] on day 1. At weeks 2, 4 or 8, participant's were up-titrated to the pediatric equivalent dose of 50 mg in adults [PED50] based on the given dose titration criteria. Following week 24, participants stayed on their individual dose level until week 52 EOS or EOT.
Participants aged 12 to < 18 years received initial dose of 25 mg of mirabegron orally once daily based on weight [PED25] on day 1. At weeks 2, 4 or 8, participant's were up-titrated to the pediatric equivalent dose of 50 mg in adults [PED50] based on the given dose titration criteria. Following week 24, participants stayed on their individual dose level until week 52 EOS or EOT.
Overall Number of Participants Analyzed 41 21
Mean (Standard Deviation)
Unit of Measure: mL
Change at Week 2 Number Analyzed 41 participants 21 participants
7.98  (101.36) 39.52  (80.24)
Change at Week 4 Number Analyzed 40 participants 20 participants
19.81  (89.04) 75.25  (105.72)
Change at Week 8 Number Analyzed 41 participants 21 participants
34.01  (89.53) 44.43  (89.01)
Change at Week 12 Number Analyzed 39 participants 21 participants
8.68  (80.16) 38.23  (66.80)
Change at Week 24 Number Analyzed 36 participants 20 participants
40.76  (116.41) 86.66  (96.55)
Change at Week 36 Number Analyzed 37 participants 21 participants
31.08  (145.63) 68.47  (122.43)
Change at Week 52 Number Analyzed 39 participants 21 participants
31.83  (94.25) 38.14  (108.06)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 2 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.617
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 7.98
Confidence Interval (2-Sided) 95%
-24.0 to 40.0
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 2 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.035
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 39.52
Confidence Interval (2-Sided) 95%
3.0 to 76.0
Estimation Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 4 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.167
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 19.81
Confidence Interval (2-Sided) 95%
-8.7 to 48.3
Estimation Comments [Not Specified]
Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 4 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.005
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 75.25
Confidence Interval (2-Sided) 95%
25.8 to 124.7
Estimation Comments [Not Specified]
Hide Statistical Analysis 5
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 8 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.020
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 34.01
Confidence Interval (2-Sided) 95%
5.7 to 62.3
Estimation Comments [Not Specified]
Hide Statistical Analysis 6
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 8 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.033
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 44.43
Confidence Interval (2-Sided) 95%
3.9 to 84.9
Estimation Comments [Not Specified]
Hide Statistical Analysis 7
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 12 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.503
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 8.68
Confidence Interval (2-Sided) 95%
-17.3 to 34.7
Estimation Comments [Not Specified]
Hide Statistical Analysis 8
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 12 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.016
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 38.23
Confidence Interval (2-Sided) 95%
7.8 to 68.6
Estimation Comments [Not Specified]
Hide Statistical Analysis 9
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 24 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.043
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 40.76
Confidence Interval (2-Sided) 95%
1.4 to 80.2
Estimation Comments [Not Specified]
Hide Statistical Analysis 10
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 24 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 86.66
Confidence Interval (2-Sided) 95%
41.5 to 131.8
Estimation Comments [Not Specified]
Hide Statistical Analysis 11
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 36 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.203
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 31.08
Confidence Interval (2-Sided) 95%
-17.5 to 79.6
Estimation Comments [Not Specified]
Hide Statistical Analysis 12
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 36 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.019
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 68.47
Confidence Interval (2-Sided) 95%
12.7 to 124.2
Estimation Comments [Not Specified]
Hide Statistical Analysis 13
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 52 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.042
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 31.83
Confidence Interval (2-Sided) 95%
1.3 to 62.4
Estimation Comments [Not Specified]
Hide Statistical Analysis 14
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 52 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.121
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 38.14
Confidence Interval (2-Sided) 95%
-11.0 to 87.3
Estimation Comments [Not Specified]
11.Secondary Outcome
Title Change From Baseline in Mean Number of Leakage Episodes Per Day
Hide Description For each participant, the mean number of leakage episodes per day (during day and night time) was calculated using all available/non-missing number of leakage episodes for the 2 measuring days in the weekend diary during day and night time. If the number of leakage episodes was recorded on 1 single day in the 7-day diary during day and night time, the mean number of leakage episodes per day during day and night time is equal to the total number of leakage episodes recorded that day during day and night time. If no leakage episodes were recorded on any day of the weekend diary during day and night time, the mean number of leakage episodes per day was zero. If nothing was recorded/diary not completed the result was missing. A valid bladder diary day in the weekend diary was any e-diary day for which >=1 catheterized volume >0 mL was recorded with complete date and time.
Time Frame Baseline and weeks 2, 4, 8, 12, 24, 36 and 52
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis population consisted of the FAS. Here, Overall Number of participants analyzed signifies number of participants evaluable for this outcome measure and number analyzed signifies number of participants with available data at each time point.
Arm/Group Title Children (3 to < 12 Years) Adolescents (12 to < 18 Years)
Hide Arm/Group Description:
Participants aged 3 to < 12 years received initial dose of 25 mg of mirabegron orally once daily based on weight [PED25] on day 1. At weeks 2, 4 or 8, participant's were up-titrated to the pediatric equivalent dose of 50 mg in adults [PED50] based on the given dose titration criteria. Following week 24, participants stayed on their individual dose level until week 52 EOS or EOT.
Participants aged 12 to < 18 years received initial dose of 25 mg of mirabegron orally once daily based on weight [PED25] on day 1. At weeks 2, 4 or 8, participant's were up-titrated to the pediatric equivalent dose of 50 mg in adults [PED50] based on the given dose titration criteria. Following week 24, participants stayed on their individual dose level until week 52 EOS or EOT.
Overall Number of Participants Analyzed 39 17
Mean (Standard Deviation)
Unit of Measure: leakage episodes per day
Change at Week 2 Number Analyzed 39 participants 17 participants
0.35  (9.35) -0.53  (1.30)
Change at Week 4 Number Analyzed 36 participants 15 participants
-1.14  (3.39) -0.87  (1.68)
Change at Week 8 Number Analyzed 35 participants 10 participants
1.16  (16.11) -0.65  (1.78)
Change at Week 12 Number Analyzed 33 participants 13 participants
0.37  (13.03) -0.65  (1.55)
Change at Week 24 Number Analyzed 31 participants 14 participants
0.18  (10.05) -0.75  (1.28)
Change at Week 36 Number Analyzed 30 participants 13 participants
-1.98  (4.33) -0.81  (1.47)
Change at Week 52 Number Analyzed 32 participants 13 participants
-0.94  (2.96) -1.12  (1.97)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 2 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.818
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 0.35
Confidence Interval (2-Sided) 95%
-2.7 to 3.4
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 2 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.114
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -0.53
Confidence Interval (2-Sided) 95%
-1.2 to 0.1
Estimation Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 4 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.052
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -1.14
Confidence Interval (2-Sided) 95%
-2.3 to 0.0
Estimation Comments [Not Specified]
Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 4 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.066
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -0.87
Confidence Interval (2-Sided) 95%
-1.8 to 0.1
Estimation Comments [Not Specified]
Hide Statistical Analysis 5
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 8 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.674
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 1.16
Confidence Interval (2-Sided) 95%
-4.4 to 6.7
Estimation Comments [Not Specified]
Hide Statistical Analysis 6
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 8 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.278
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -0.65
Confidence Interval (2-Sided) 95%
-1.9 to 0.6
Estimation Comments [Not Specified]
Hide Statistical Analysis 7
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 12 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.871
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 0.37
Confidence Interval (2-Sided) 95%
-4.2 to 5.0
Estimation Comments [Not Specified]
Hide Statistical Analysis 8
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 12 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.153
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -0.65
Confidence Interval (2-Sided) 95%
-1.6 to 0.3
Estimation Comments [Not Specified]
Hide Statistical Analysis 9
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 24 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.922
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 0.18
Confidence Interval (2-Sided) 95%
-3.5 to 3.9
Estimation Comments [Not Specified]
Hide Statistical Analysis 10
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 24 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.047
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -0.75
Confidence Interval (2-Sided) 95%
-1.5 to 0.0
Estimation Comments [Not Specified]
Hide Statistical Analysis 11
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 36 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.018
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -1.98
Confidence Interval (2-Sided) 95%
-3.6 to -0.4
Estimation Comments [Not Specified]
Hide Statistical Analysis 12
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 36 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.070
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -0.81
Confidence Interval (2-Sided) 95%
-1.7 to 0.1
Estimation Comments [Not Specified]
Hide Statistical Analysis 13
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 52 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.083
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -0.94
Confidence Interval (2-Sided) 95%
-2.0 to 0.1
Estimation Comments [Not Specified]
Hide Statistical Analysis 14
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 52 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.064
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -1.12
Confidence Interval (2-Sided) 95%
-2.3 to 0.1
Estimation Comments [Not Specified]
12.Secondary Outcome
Title Change From Baseline in Number of Dry Days Per 7 Days (Day and Night Time)
Hide Description Dry days were defined as leakage-free days, this included day and night time. Participants recorded dry days in the 7-day diary. Dry days were calculated as follows: Ddry was the number of valid diary days where the response to the question 'Did you leak between this catheterization and the last one' was 'No' each time a new catheterization was entered in the e-diary during the day & night time period. Dwet was the number of valid diary days where the response to the question 'Did you leak between this catheterization and the last one' was 'Yes' for at least one catheterization entered during the day and night time period. If (Ddry + Dwet) > 3, the number of dry days per 7 days was calculated as Ddry/(Ddry + Dwet) x 7, otherwise the value was missing.
Time Frame Baseline and weeks 2, 4, 8, 12, 24, 36 and 52
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis population consisted of the FAS. Here, Overall Number of participants analyzed signifies number of participants evaluable for this outcome measure and number analyzed signifies number of participants with available data at each time point.
Arm/Group Title Children (3 to < 12 Years) Adolescents (12 to < 18 Years)
Hide Arm/Group Description:
Participants aged 3 to < 12 years received initial dose of 25 mg of mirabegron orally once daily based on weight [PED25] on day 1. At weeks 2, 4 or 8, participant's were up-titrated to the pediatric equivalent dose of 50 mg in adults [PED50] based on the given dose titration criteria. Following week 24, participants stayed on their individual dose level until week 52 EOS or EOT.
Participants aged 12 to < 18 years received initial dose of 25 mg of mirabegron orally once daily based on weight [PED25] on day 1. At weeks 2, 4 or 8, participant's were up-titrated to the pediatric equivalent dose of 50 mg in adults [PED50] based on the given dose titration criteria. Following week 24, participants stayed on their individual dose level until week 52 EOS or EOT.
Overall Number of Participants Analyzed 43 24
Mean (Standard Deviation)
Unit of Measure: dry days per 7 days
Change at Week 2 Number Analyzed 43 participants 24 participants
0.34  (0.91) 0.82  (1.90)
Change at Week 4 Number Analyzed 42 participants 24 participants
0.68  (1.69) 1.36  (1.91)
Change at Week 8 Number Analyzed 43 participants 23 participants
1.14  (2.15) 2.26  (2.48)
Change at Week 12 Number Analyzed 43 participants 24 participants
1.31  (2.50) 1.93  (2.46)
Change at Week 24 Number Analyzed 41 participants 24 participants
1.34  (2.18) 2.17  (2.38)
Change at Week 36 Number Analyzed 39 participants 24 participants
1.33  (2.43) 1.88  (2.13)
Change at Week 52 Number Analyzed 40 participants 24 participants
1.38  (2.65) 2.14  (2.51)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 2 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.018
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 0.34
Confidence Interval (2-Sided) 95%
0.1 to 0.6
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 2 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.045
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 0.82
Confidence Interval (2-Sided) 95%
0.0 to 1.6
Estimation Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 4 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.013
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 0.68
Confidence Interval (2-Sided) 95%
0.1 to 1.2
Estimation Comments [Not Specified]
Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 4 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.002
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 1.36
Confidence Interval (2-Sided) 95%
0.6 to 2.2
Estimation Comments [Not Specified]
Hide Statistical Analysis 5
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 8 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.001
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 1.14
Confidence Interval (2-Sided) 95%
0.5 to 1.8
Estimation Comments [Not Specified]
Hide Statistical Analysis 6
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 8 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 2.26
Confidence Interval (2-Sided) 95%
1.2 to 3.3
Estimation Comments [Not Specified]
Hide Statistical Analysis 7
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 12 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.001
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 1.31
Confidence Interval (2-Sided) 95%
0.5 to 2.1
Estimation Comments [Not Specified]
Hide Statistical Analysis 8
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 12 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 1.93
Confidence Interval (2-Sided) 95%
0.9 to 3.0
Estimation Comments [Not Specified]
Hide Statistical Analysis 9
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 24 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 1.34
Confidence Interval (2-Sided) 95%
0.7 to 2.0
Estimation Comments [Not Specified]
Hide Statistical Analysis 10
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 24 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 2.17
Confidence Interval (2-Sided) 95%
1.2 to 3.2
Estimation Comments [Not Specified]
Hide Statistical Analysis 11
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 36 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.002
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 1.33
Confidence Interval (2-Sided) 95%
0.5 to 2.1
Estimation Comments [Not Specified]
Hide Statistical Analysis 12
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 36 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 1.88
Confidence Interval (2-Sided) 95%
1.0 to 2.8
Estimation Comments [Not Specified]
Hide Statistical Analysis 13
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 52 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.002
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 1.38
Confidence Interval (2-Sided) 95%
0.5 to 2.2
Estimation Comments [Not Specified]
Hide Statistical Analysis 14
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 52 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 2.14
Confidence Interval (2-Sided) 95%
1.1 to 3.2
Estimation Comments [Not Specified]
13.Secondary Outcome
Title Change From Baseline in Pediatric Incontinence Questionnaire (PIN-Q) Score
Hide Description PIN-Q measured quality of life via an e-diary. Total score ranged from 0 (no effect) to 80 (worst effect); decrease in score indicated improvement. Total score was 20x average of individual PinQ items, the 20 Likert scales were converted to a score: Items 6 & 17; 0: "No" to 4: "Definitely" was used; & For the other 18 items; 0: "No" to 4: "All the time" was used. Expectation that questionnaires had limited missing values; if answers >2 questions were missing, total score was not calculated & was missing. Individual item scores were directly imputed. Change from baseline to each post-baseline visit in the total score was post-baseline visit value minus baseline value. If either baseline or post-baseline visit value was missing, change from baseline was missing. If change was: <0, improvement between 2 time-points; =0, no change between 2 time points; >0, worsening between 2 time points.
Time Frame Baseline and weeks 24 and 52
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis population consisted of the FAS. Here, Overall Number of participants analyzed signifies number of participants evaluable for this outcome measure and number analyzed signifies number of participants with available data at each time point.
Arm/Group Title Children (3 to < 12 Years) Adolescents (12 to < 18 Years)
Hide Arm/Group Description:
Participants aged 3 to < 12 years received initial dose of 25 mg of mirabegron orally once daily based on weight [PED25] on day 1. At weeks 2, 4 or 8, participant's were up-titrated to the pediatric equivalent dose of 50 mg in adults [PED50] based on the given dose titration criteria. Following week 24, participants stayed on their individual dose level until week 52 EOS or EOT.
Participants aged 12 to < 18 years received initial dose of 25 mg of mirabegron orally once daily based on weight [PED25] on day 1. At weeks 2, 4 or 8, participant's were up-titrated to the pediatric equivalent dose of 50 mg in adults [PED50] based on the given dose titration criteria. Following week 24, participants stayed on their individual dose level until week 52 EOS or EOT.
Overall Number of Participants Analyzed 24 21
Mean (Standard Deviation)
Unit of Measure: units on a scale
Change at Week 24 Number Analyzed 24 participants 21 participants
2.04  (10.53) -4.90  (14.13)
Change at Week 52 Number Analyzed 23 participants 19 participants
1.30  (12.17) -6.79  (14.50)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 24 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.352
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 2.04
Confidence Interval (2-Sided) 95%
-2.40 to 6.49
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 24 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.127
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -4.90
Confidence Interval (2-Sided) 95%
-11.34 to 1.53
Estimation Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 52 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.613
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 1.30
Confidence Interval (2-Sided) 95%
-3.96 to 6.57
Estimation Comments [Not Specified]
Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 52 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.056
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -6.79
Confidence Interval (2-Sided) 95%
-13.78 to 0.20
Estimation Comments [Not Specified]
14.Secondary Outcome
Title Change From Baseline in Patient Global Impression of Severity Scale (PGI-S)
Hide Description The PGI-S was an answer to the question: "How did you feel about your bladder condition during the past 3 days?" Participants evaluated their recent condition as "Really Bad"(0), "Bad" (1), "Not Bad, Not Good" (2), "Good" (3) &"Really Good" (4). An increase indicated improvement. The total score ranged from 0 to 4, where higher scores indicated improvement.The change from baseline to each postbaseline visit in the PGI-S score is the value at the post-baseline visit minus the value at the baseline visit. If either the baseline or the post-baseline visit value is missing, the change from baseline was missing. A positive change indicated an improvement while a negative change indicated a worsening.
Time Frame Baseline and weeks 24 and 52
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis population consisted of the FAS. Here, Overall Number of participants analyzed signifies number of participants evaluable for this outcome measure and number analyzed signifies number of participants with available data at each time point.
Arm/Group Title Children (3 to < 12 Years) Adolescents (12 to < 18 Years)
Hide Arm/Group Description:
Participants aged 3 to < 12 years received initial dose of 25 mg of mirabegron orally once daily based on weight [PED25] on day 1. At weeks 2, 4 or 8, participant's were up-titrated to the pediatric equivalent dose of 50 mg in adults [PED50] based on the given dose titration criteria. Following week 24, participants stayed on their individual dose level until week 52 EOS or EOT.
Participants aged 12 to < 18 years received initial dose of 25 mg of mirabegron orally once daily based on weight [PED25] on day 1. At weeks 2, 4 or 8, participant's were up-titrated to the pediatric equivalent dose of 50 mg in adults [PED50] based on the given dose titration criteria. Following week 24, participants stayed on their individual dose level until week 52 EOS or EOT.
Overall Number of Participants Analyzed 25 22
Mean (Standard Deviation)
Unit of Measure: units on a scale
Change at Week 24 Number Analyzed 25 participants 22 participants
0.36  (1.22) 0.64  (1.00)
Change at Week 52 Number Analyzed 24 participants 19 participants
0.42  (1.21) 0.95  (1.18)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 24 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.153
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 0.36
Confidence Interval (2-Sided) 95%
-0.14 to 0.86
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 24 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.007
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 0.64
Confidence Interval (2-Sided) 95%
0.19 to 1.08
Estimation Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Children (3 to < 12 Years)
Comments Change From Baseline at Week 52 - Children
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.106
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 0.42
Confidence Interval (2-Sided) 95%
-0.10 to 0.93
Estimation Comments [Not Specified]
Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection Adolescents (12 to < 18 Years)
Comments Change From Baseline at Week 52 - Adolescents
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.003
Comments From a 2-sided paired t-test, testing the null hypothesis that change from baseline is equal to 0.
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 0.95
Confidence Interval (2-Sided) 95%
0.38 to 1.51
Estimation Comments [Not Specified]
15.Secondary Outcome
Title Number of Participants With Clinician Global Impression of Change (CGI-C)
Hide Description The Clinician Global Impression of Change (CGI-C) is a 7 point scale that required the clinician to assess how much the participant's overall bladder symptoms since the start of the study on day 1 has improved or worsened and rated as: very much improved (1); much improved (2); minimally improved (3); no change (4); minimally worse (5); much worse (6); or very much worse (7). The total score range from 1-7, where lower scores indicated improvement.
Time Frame Weeks 24 and 52
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis population consisted of the FAS. Here, Overall Number of participants analyzed signifies number of participants evaluable for this outcome measure and number analyzed signifies number of participants with available data at each time point.
Arm/Group Title Children (3 to < 12 Years) Adolescents (12 to < 18 Years)